Events9th International Electronic Conference on Medicinal Chemistry
Published
with-doi10.3390/ECMC2023-15572 (registering DOI)
This submission belongs to the session S3. General of the event 9th International Electronic Conference on Medicinal Chemistry
Published date
01 Nov, 2023
Academic Editor
author-avatarAlfredo Berzal-Herranz
Citation
Haya Al Hilal, Anton Kiselev, Marianna Maretina, Anna Egorova, Assessment of nuclear gems quantity as a potential biomarker for evaluating the efficacy of drugs for spinal muscular atrophy., in Proceedings of 9th International Electronic Conference on Medicinal Chemistry, 1 November–30 November 2023, MDPI: Basel, Switzerland, doi: 10.3390/ECMC2023-15572
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Assessment of nuclear gems quantity as a potential biomarker for evaluating the efficacy of drugs for spinal muscular atrophy.

Anna Egorova 2
image
1. 1 Department of Genomic Medicine named after V.S. Baranov, D.O. Ott Research Institute of Obstetrics, Gynecology and Reproductology, Mendeleevskaya Line 3, 199034 Saint-Petersburg, Russia; 2 Faculty of Biology, Saint-Petersburg State University, Univers
2. Department of Genomic Medicine named after V.S. Baranov, D.O. Ott Research Institute of Obstetrics, Gynecology and Reproductology, Mendeleevskaya Line 3, 199034 Saint-Petersburg, Russia;
Abstract
Keywords
spinal muscular atrophy
SMN1 gene
SMN2 gene
nuclear gems
antisense oligonucleotides
splicing correction.